New Gene Transfer Strategy Shows Promise for Limb Girdle and Other Muscular Dystrophies, The Research Institute of Nationwide Children's Hospital Study

7/9/2012 7:44:49 AM

The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's Hospital study describes a new gene therapy approach capable of delivering full-length versions of large genes and improving skeletal muscle function. The strategy may hold new hope for treating dysferlinopathies and other muscular dystrophies.